Transfer of the ADA Gene into Bone Marrow Cells and Peripheral Blood Lymphocytes for the Treatment of Patients Affected by ADA-Deficient SCID

Author:

Bordignon Claudio1,Mavilio Fulvio2,Ferrari Giuliana2,Servida Paolo2,Ugazio Alberto G.3,Notarangelo Luigi D.3,Gilboa Eli4,Rossini Silvano2,O'Reilly Richard J.4,Smith Clayton A.4,Gillio Alfred P.4,Anderson W. French5,Blaese R. Michael6,Moen Robert C.7,Eglitis Martin A.7

Affiliation:

1. Head, Bone Marrow Transplantation and Gene Therapy Program, Istituto Scientifico H.S. Raffaele, via Olgettina 60, 20132 Milano, Italy. Telephone: 39-2-26432357; Telefax: 39-2-26432285.

2. Istituto Scientifico H.S. Raffaele.

3. Clinica Pediatrica, Università di Brescia.

4. Memorial Sloan-Kettering Cancer Center.

5. Chief, Molecular Hematology Branch, NHLBI.

6. Chief, Cellular Immunology Section, Metabolism Branch, NCI.

7. Genetic Therapy Inc.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Cited by 52 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. Updated Management Guidelines for Adenosine Deaminase Deficiency;The Journal of Allergy and Clinical Immunology: In Practice;2023-06

2. Review of Treatment for Adenosine Deaminase Deficiency (ADA) Severe Combined Immunodeficiency (SCID);Therapeutics and Clinical Risk Management;2022-09

3. Targeted genome editing for the correction or alleviation of primary Immunodeficiencies;Progress in Molecular Biology and Translational Science;2021

4. Gene therapy for primary immunodeficiency;Human Molecular Genetics;2019-07-11

5. Terapias avanzadas en enfermedades raras;Arbor;2018-11-30

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